BUSM researcher receives two NIH grants to study scleroderma

Boston University School of Medicine (BUSM) researcher Robert Lafyatis, MD, recently was awarded two grants from the National Institutes of Health's (NIH) National Institute of Arthritis and Musculoskeletal and Skin Diseases. A 5-year, $8 million Centers of Research Translation (CORT) (P50) grant as well as a 5-year $3.3 million (P30) grant. Funding from both grants will be used to study systemic sclerosis (SSc) also known as scleroderma, a rare and complex rheumatic disease involving widespread scarring and vascular disease within multiple organ systems.

SSc remains one of the most difficult rheumatic diseases to manage, with limited effective therapies. These projects were designed to coordinate multiple scientists and clinicians to accelerate the understanding of the disease process through interactive patient-oriented studies into markers of disease activity, investigation of pathogenesis and trials of novel therapeutics.

The P50 grant will fund clinical research, identifying new paradigms for early phase clinical trials and molecular research into scleroderma pathogenesis. The P30 will allow the researchers at four sites: BUSM, University of Pittsburg Medical Center, Northwestern School of Medicine and Dartmouth Medical Center, to set up core services for scleroderma research, with the ultimate goal being a national resource for investigators studying scleroderma across the country.

One of the greatest impediments to finding new treatments is the heterogeneity of patient presentation and disease progression. Clinical markers are unable to predict onset and/or progression of the major complications, such as progressive fibrotic skin disease, pulmonary arterial hypertension and interstitial lung disease, each seen in a minority of SSc patients.

According to the researchers, identification of biomarkers permitting early recognition of these complications would potentially permit more targeted therapies, but also provide "at risk" populations for enrolling in therapeutic trials.

"Empowered by a very large SSc clinical population, we propose careful clinical evaluations, coupled with robust molecular approaches to identify skin, serum and peripheral blood mononuclear cell disease biomarkers," explained principal investigator Lafyatis, who also is a professor of medicine at BUSM.